Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

PHASE3CompletedINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

April 10, 2019

Primary Completion Date

January 27, 2023

Study Completion Date

January 27, 2023

Conditions
Arginase I DeficiencyHyperargininemia
Interventions
DRUG

Pegzilarginase

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

DRUG

Placebo

Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated

Trial Locations (32)

10029

Icahn School of Medicine at Mount Sinai, New York

11040

Cohen Children's Medical Center (Northwell Health), Queens

15224

University of Pittsburgh Medical Center, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

20010

Children's National Medical Center, Washington D.C.

27710

Duke University Medical Center, Durham

30322

Emory University, Atlanta

32610

University of Florida College of Medicine, Gainesville

37232

Vanderbilt University Medical Center, Nashville

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

72758

Harvey Pediatrics, Rogers

75390

UT Southwestern Medical Center, Dallas

77030

University of Texas Health Science Center Medical School at Houston, Houston

84108

University of Utah Hospitals & Clinics, Salt Lake City

92868

Children's Hospital of Orange County, Orange

94305

Stanford University School of Medicine, Stanford

98105

Seattle Children's Hospital, Seattle

Unknown

LKH Bregenz, Bregenz

Medizinische Universität Innsbruck, Innsbruck

McGill University Health Center, Montreal

Hôpital Necker - Enfants Malades, Paris

Hopital des Enfants, Talence

Universitaetsklinikum Muenster, Münster

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz, Mainz

Fondazione MBBM, Monza

Ospedale Pediatrico Bambino Gesù, Roma

Azienda Ospedaliera Città della Salute e della Scienza di Torino, Torino

Birmingham Children's Hospital, Birmingham

University Hospital of Wales, Cardiff

Great Ormond Street Hospital for Children, London

Willink Biochemical Genetics Unit, Manchester

Salford Royal, Salford

Sponsors
All Listed Sponsors
lead

Aeglea Biotherapeutics

INDUSTRY

NCT03921541 - Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency | Biotech Hunter | Biotech Hunter